Background Hepatitis-associated aplastic anemia is a variant of aplast
ic anemia in which aplastic anemia follows an acute attack of hepatiti
s. The aplastic anemia, however, is often fatal if untreated. To chara
cterize the illness, investigate the role of hepatitis viruses, and as
sess the response to immunosuppressive treatment, we studied patients
with the syndrome who were referred to the National Institutes of Heal
th (NIH). Methods Standard hematologic and biochemical tests and measu
rements of bone marrow cellularity were used to monitor the patients'
response to treatment. Serum was assayed for antibodies and antigens r
elated to hepatitis A, B, and C viruses and for the RNA of hepatitis C
and GB virus C by the polymerase chain reaction. All patients were tr
eated with antithymocyte globulin and cyclosporine. Results Ten patien
ts with hepatitis-associated aplastic anemia were referred to the NIH
between 1990 and 1996; all had the typical features of this syndrome.
There was evidence of activated CD8 T lymphocytes in the blood. Serolo
gic tests for hepatitis A, B, and C viruses were negative; RNA of hepa
titis C virus was undetectable in all patients, but RNA of GB virus C
was detected in three patients. Seven of the patients responded to int
ensive immunosuppressive treatment; the three who did not respond all
died within one year of treatment, two from complications of stem-cell
or marrow transplantation. Conclusions The hepatitis of the hepatitis
-associated aplastic anemia does not appear to be caused by any of the
known hepatitis viruses. We recommend immunosuppressive treatment for
patients who do not have an HLA-matched related donor available for b
one marrow transplantation. Several features of the syndrome suggest t
hat it is mediated by immunopathologic mechanisms. (C) 1997, Massachus
etts Medical Society.